A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care

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Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
Phase IIISupportive care, TreatmentCompleted18 and overPharmaceutical / IndustryAZA PH GL 2003 CL 001

Trial Description


The purpose of this study is to determine whether patients with high-risk myelodysplastic syndromes (MDS) treated with azacitidine have improved survival compared to conventional care treatments. The study will also assess the effect of treatments on response, duration of response, and transformation to acute myeloid leukemia (AML). The study will continue for 12 months following last patient enrolled.

See study AZA PH GL 2003 CL 001 E for information about the extension to this study.

Further Study Information

Comparison/Control Interventions offered the physician three options:

  • Best supportive care (BSC) alone,
  • Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or
  • Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle).

All three options included best supportive care. Neither the experimental group (azacitidine) nor any of the comparison/control options allowed use of erythropoietin.

Duration of Intervention: Patients will be treated until death, withdrawal, unacceptable toxicity or conclusion of the study.

Eligibility Criteria

Inclusion Criteria:

  • Have a diagnosis of refractory anemia with excess blasts or refractory anemia with excess blasts in transformation according to the French-American-British classification system for myelodysplastic syndromes (MDS) and a relatively high risk of acute myeloid leukemia (AML) transformation, with an International Prognostic Scoring System score of INT-2 or High.
  • Be 18 years of age or older
  • Have a life expectancy of at least 3 months
  • Be unlikely to proceed to bone marrow or stem cell transplantation therapy following remission
  • Have serum bilirubin levels less than or equal to 1.5 times the upper limit of normal range for the laboratory
  • Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to 2 times the upper limit of normal (unless these are considered to be related to transfusion-induced secondary hemosiderosis)
  • Have serum creatinine levels less than or equal to 1.5 times the upper limit of normal

Exclusion Criteria:

  • Secondary myelodysplastic syndromes (MDS)
  • Prior treatment with azacitidine;
  • Prior history of acute myeloid leukemia (AML);
  • Malignant disease diagnosed within prior 12 months;
  • Metastatic disease;
  • Hepatic tumors;
  • Radiation, chemotherapy, cytotoxic therapy for non-MDS conditions within prior 12 months;
  • Prior transplantation or cytotoxic therapy to treat MDS;
  • Serious medical illness likely to limit survival to 12 months or less;
  • Treatment with erythropoietin or myeloid growth factors during prior 21 days or androgenic hormones during prior 13 days;
  • Active HIV, viral hepatitis type B or C;
  • Treatment with investigational drugs during prior 30 days;
  • Within the 28-day screening period, documented red cell folate deficiency, as evidenced by red blood cell folate (not serum folate) or vitamin B12 deficiency

Trial Contact Information

Trial Lead Organizations/Sponsors

Celgene Corporation

    CL Beach, Study Director

    Link to the current ClinicalTrials.gov record.
    NLM Identifier NCT00071799
    ClinicalTrials.gov processed this data on May 15, 2015

    Note: Information about this trial is from the ClinicalTrials.gov database. The versions designated for health professionals and patients contain the same text. Minor changes may be made to the ClinicalTrials.gov record to standardize the names of study sponsors, sites, and contacts. Cancer.gov only lists sites that are recruiting patients for active trials, whereas ClinicalTrials.gov lists all sites for all trials. Questions and comments regarding the presented information should be directed to ClinicalTrials.gov.